Abstract: The present disclosure relates to methods for targeted genome editing in eukaryotic cells. In some embodiments, methods may comprise preparing an mRNA encoding a Cas9 protein and preparing a guide RNA, wherein the guide RNA comprises a crRNA and tracrRNA. Methods may further comprise introducing the mRNA and guide RNA into a eukaryotic cell, such as a non-human embryo, wherein the mRNA induces expression of the Cas9 protein in the non-human embryo, and wherein the Cas9 protein and guide RNA induce a modification at a target endogenous nucleic acid sequence. Methods may further comprise transferring the genome modified embryo into a foster mother and allowing the foster mother to produce a F0 animal having the modification at the target endogenous nucleic acid sequence.

Abstract: The present disclosure relates to methods for producing a genome modified non-human embryo by inducing modifications at target endogenous nucleic acid sequences. For example, methods disclosed herein may comprise providing a Cas9/sgRNA complex, comprising a Cas9 protein and a single-guide RNA (sgRNA) in a cell-free buffer. Methods may further comprise introducing the Cas9/sgRNA complex into a non-human embryo, wherein the Cas9/sgRNA complex induces a modification at a target endogenous nucleic acid of the non-human embryo to provide for a genome modified embryo. Methods may further comprise transferring the genome modified embryo into a foster mother and allowing the foster mother to produce a F0 animal having the modification at the target endogenous nucleic acid.

Abstract: The present disclosure relates to methods for producing a genome modified non-human embryo by inducing modifications at target endogenous nucleic acid sequences. For example, methods disclosed herein may comprise providing a Cas9/sgRNA complex, comprising a Cas9 protein and a single-guide RNA (sgRNA) in a cell-free buffer. Methods may further comprise introducing the Cas9/sgRNA complex into a non-human embryo, wherein the Cas9/sgRNA complex induces a modification at a target endogenous nucleic acid of the non-human embryo to provide for a genome modified embryo. Methods may further comprise transferring the genome modified embryo into a foster mother and allowing the foster mother to produce a F0 animal having the modification at the target endogenous nucleic acid.

Abstract: The present disclosure relates to in vitro and ex vivo methods for cleaving a target DNA in a mammalian cell, inducing a targeted mutagenesis in a mammalian cell, and/or genome editing in a mammalian cell. Methods may comprise providing a composition comprising a single-chain guide RNA (sgRNA) and a Cas9 protein, and transferring the composition into the cell, wherein the Cas9 protein is a Streptococcus pyogenes Cas9 protein. In some embodiments, the mammalian cell may be a human cell.

Abstract: The present invention relates to an artificially engineered CRISPR/Cas9 system. More particularly, the present invention relates to an artificially engineered CRISPR enzyme having enhanced target specificity and a use of an artificially engineered CRISPR/Cas9 system including the same enzyme in genome and/or epigenome manipulation or modification, genome targeting, genome editing, and in vitro diagnosis, etc.

Abstract: The present invention relates to a hemophilia B rat and a method of producing the hemophilia B rat. More particularly, the present invention relates to a hemophilia B rat having F9 factor knocked-down or knocked-out and a method of producing the hemophilia B rat.

Abstract: The present invention relates to an artificially engineered CRISPR/Cas9 system. More particularly, the present invention relates to an artificially engineered CRISPR enzyme having enhanced target specificity and a use of an artificially engineered CRISPR/Cas9 system including the same enzyme in genome and/or epigenome manipulation or modification, genome targeting, genome editing, and in vitro diagnosis, etc.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. The composition may comprise a single-chain guide RNA (sgRNA) and a Streptococcus pyogenes Cas9 protein. In some embodiments, the sgRNA and the Cas9 protein may be present in a molar ratio ranging from 29:14.0 to 29:1.4.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, methods disclosed herein may comprise preparing a Cas9/RNA complex, wherein the Cas9/RNA complex comprises a Cas9 protein and a guide RNA. Methods may further comprise introducing the Cas9/RNA complex into a non-human embryo, wherein the Cas9/RNA complex induces a modification at a target endogenous nucleic acid of the non-human embryo to provide for a genome modified embryo. Methods may further comprise transferring the genome modified embryo into a foster mother and allowing the foster mother to produce a F0 animal having the modification at the target endogenous nucleic acid.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, disclosed herein is a method of producing an engineered eukaryotic cell. In some embodiments, the method may comprise preparing a Cas9 protein, preparing a sgRNA, and preparing a cell-free buffer. The method may further comprise disposing the Cas9 protein and sgRNA in the cell-free buffer to provide for a transfection mixture and transfecting the transfection mixture into a eukaryotic cell, wherein a Cas9/sgRNA complex formed by the Cas9 protein and the sgRNA induces a modification of a target endogenous DNA sequence in the nucleus of the eukaryotic cell to provide for an engineered eukaryotic cell.

Abstract: The present invention relates to an artificially manipulated unsaturated fatty acid biosynthesis-associated factor and use thereof to increase the content of a specific unsaturated fatty acid of a plant body. More particularly, the present invention relates to a system capable of artificially controlling unsaturated fatty acid biosynthesis and a plant body produced thereby, which include an artificially manipulated unsaturated fatty acid biosynthesis-associated factor to control unsaturated fatty acid biosynthesis and a composition capable of artificially manipulating the factor. In a specific aspect, the present invention relates to artificially manipulated unsaturated fatty acid biosynthesis-associated factors such as FAD2, FAD3, FAD6, FAD7 and FAD8 and/or an unsaturated fatty acid biosynthesis controlling system by an expression product thereof.

Abstract: Provided is a platform for expressing a protein of interest by artificially manipulating the liver, and more particularly, to a platform for alleviating or treating a genetic disorder or improving a body function by inducing expression by inserting a transgene (e.g., a therapeutic gene) which can function or be expressed normally, into a high-expression secretory gene, instead of a disease gene which functions or is expressed abnormally. The high-expression secretory gene includes the HP or APOC3 gene. The transgene includes one that is highly expressed using a promoter in a hepatocyte genome and is secretory out of the cell.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for Cas9/RNA complexes that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. The Cas9/RNA complex may comprise a recombinant Cas9 protein including a nuclear localization signal (NLS) and a guide RNA including a crRNA and a tracrRNA. The Cas9/RNA complex may be a combination of the recombinant Cas9 protein and the guide RNA. The guide RNA may be transcribed in vitro or synthesized chemically. The target endogenous nucleic acid sequence may include a portion complementary to the crRNA of the guide RNA.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, disclosed herein is a method of producing an engineered eukaryotic cell. In some embodiments, the method may comprise preparing a Cas9 protein, preparing a sgRNA, and preparing a cell-free buffer. The method may further comprise disposing the Cas9 protein and sgRNA in the cell-free buffer to provide for a transfection mixture and transfecting the transfection mixture into a eukaryotic cell, wherein a Cas9/sgRNA complex formed by the Cas9 protein and the sgRNA induces a modification of a target endogenous DNA sequence in the nucleus of the eukaryotic cell to provide for an engineered eukaryotic cell.

Abstract: The present invention relates to an artificially engineered CRISPR/Cas9 system. More particularly, the present invention relates to an artificially engineered CRISPR enzyme having enhanced target specificity and a use of an artificially engineered CRISPR/Cas9 system including the same enzyme in genome and/or epigenome manipulation or modification, genome targeting, genome editing, and in vitro diagnosis, etc.

Abstract: The present invention relates to an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder, and to a use thereof. More specifically, the present invention relates to a system capable of performing artificial SC function control and/or treating or alleviating a disease due to an SC function disorder, the system comprising: an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder; and/or a composition for treating or alleviating a disease due to an SC function disorder. In a specific aspect, the present invention relates to an SC function-controlling system by an SC function-controlling factor, such as artificially manipulated PMP22, and/or an expression product thereof.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, methods disclosed herein may comprise preparing a Cas9/RNA complex, wherein the Cas9/RNA complex comprises a Cas9 protein and a guide RNA. Methods may further comprise introducing the Cas9/RNA complex into a non-human embryo, wherein the Cas9/RNA complex induces a modification at a target endogenous nucleic acid of the non-human embryo to provide for a genome modified embryo. Methods may further comprise transferring the genome modified embryo into a foster mother and allowing the foster mother to produce a F0 animal having the modification at the target endogenous nucleic acid.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells.

Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, disclosed herein is a system for inducing targeted disruption of endogenous genes in a eukaryotic cell, the system comprising: an extracellular Cas9/RNA complex; cell-free and a buffer; wherein the extracellular Cas9/RNA complex comprises: a recombinant Cas9; and a guide RNA having a CRISPR RNA (crRNA) and a transactivating crRNA (tracrRNA); wherein the extracellular Cas9/RNA complex is disposed in the cell-free buffer and wherein the extracellular Cas9/RNA complex is complexed prior to being introduced into the eukaryotic cell; and wherein the Cas9/RNA complex functions as an endonuclease that induces targeted disruption of the target DNA upon introduction into the eukaryotic cell.